Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.

Mucopolysaccharidosis type VII (MPS VII; Sly syndrome) is one of a group of lysosomal storage diseases that share many clinical features, including mental retardation and hearing loss. Lysosomal storage in neurons of the brain and the associated behavioral abnormalities characteristic of a murine mo...

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Bibliografische gegevens
Gepubliceerd in:J Clin Invest
Hoofdauteurs: O'Connor, L H, Erway, L C, Vogler, C A, Sly, W S, Nicholes, A, Grubb, J, Holmberg, S W, Levy, B, Sands, M S
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society for Clinical Investigation 1998
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Research Article
Online toegang:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC508717/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/9525982/
https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI1773
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